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The company’s intein-based technology is initially aimed at Stargardt disease, a type of macular degeneration.
FEATURED STORIES
As obesity drug developers compete for the highest weight-loss efficacy, experts contend that overall health outcomes—evidenced by successful studies in therapeutic areas like cardiovascular and sleep apnea—may prove a greater market advantage.
Facing declining valuations and funding challenges, public biotechs like bluebird bio are going private to restructure, reduce regulatory burdens and refocus on long-term growth.
Johnson & Johnson has been fighting thousands of lawsuits over its now-discontinued talc products for 16 years. A pending judge’s ruling could finally put the issue to bed once and for all.
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One year since forming its Advisory Board, Lokavant, the clinical trial intelligence platform company, announces three new members, including Pfizer’s Head of Predictive Analytics Jonathan Crowther.
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When talking to some of the most impressive women in biopharma, the conversation inevitably turned to what these women wanted other entrepreneurs to know. Here’s the best of the best of that advice.
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A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Govorestat failed to meet its primary endpoint in a Phase II/III trial for a rare form of Charcot-Marie-Tooth disease, a few months after the FDA rebuffed the same drug in a similar indication.
Regeneron promised to comply with 23andMe’s consumer privacy policies and related data security laws.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
The restrictions on Novavax’s vaccine could portend changes at the FDA. Commissioner Marty Makary suggested last week that the agency could update its vaccine approval guidelines “in the coming days.”
Analysts at BMO Capital Markets said in a weekend note that a non-invasive blood test could help boost uptake of Alzheimer’s disease therapies.
The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain.
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
While the Trump administration has painted the jettisoning of staff and regulations as good for business, there are multiple reasons it’s unlikely to work out that way.
The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.